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Research Studies Finder

Research Studies Finder

Use this finder to find a study or trial that is the right fit for your child. Also, filter results by phase — which are the specific steps researchers take throughout the trial to find answers, solutions and cures.

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Showing 1 - 10 of 366 results

Tiragolumab and Atezolizumab in SMARCB1 or SMARCA4 Deficient Tumors

This study enrolls patients that have been diagnosed with SMARCB1 or SMARCB4 deficient cancer that has either come back (relapsed) or does not respond to therapy (is refractory) or have been newly diagnosed with a SMARCB1 or SMARCB4 deficient cancer that has no known standard treatment. This study involves taking study drugs called tiragolumab and atezolizumab. These drugs are a type of immunotherapy that try to make the immune system fight cancer. The purpose of the study is to learn more about the safety of tiragolumab in children and to see how well the combination of tiragolumab and atezolizumab works when given to children and adults with these types of tumors. This study has two parts. In Part A, participants will receive tiragolumab. In Part B, participants will receive both tiragolumab and atezolizumab.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Central Nervous System (CNS) Germ Cell Tumors

Neurofibromatosis 1 Optic Glioma's - Understanding the mechanisms of Vision Loss for Visual Restoration Strategies

In this study we are seeking to learn how different eye tests and devices can help understand vision in patients with NF1, including how vision loss might show up on different eye imaging devices that measure the nerves or the blood vessels of the eye. The study aims to understand the mechanisms of vision loss caused by neurofibromatosis type 1 (NF1)-associated optic pathway gliomas, or NF1-OPGs. Subjects eight years and older with NF1 are eligible to enroll.
Subjects may not benefit directly from participating in this study, but the knowledge gained will help doctors understand the potential benefit of using these tests and other procedures to improve their ability to identify the best tests to monitor visual function, and ultimately identify treatments to improve vision, in children and adults with NF1.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

Conditions: Neurofibromatosis type 1

Neurophysiological Biomarkers in Rett Syndrome

The purpose of this study to learn more about the brain function of females with Rett Syndrome and how it compares to the brain function of typically developing females. The primary investigator of the study is Dr. Eric Marsh. For our research to be successful we need to partner with families who have a typically developing daughter between the age of 1 and 17. If your child agrees to help us with our study she will be asked to:

  • Complete intelligence testing
  • Receive an Evoked Potential measurement - this is a non-invasive procedure similar to an EEG.
  • Complete yearly follow up visits
All participants are compensated for their time and travel.

Phase: N/A

Actively recruiting: Yes

Category: Children, Healthy Controls

Therapeutic Role Playing Games for Autistic Individuals

Research has shown that table top role playing games (RPGs) like Dungeons & Dragons can help autistic people, as the game allows people to practice social skills and interaction in a safe environment while having fun. The goal of this new research project is to determine how aware parents and providers are that games like Dungeons & Dragons can help and what their experience is like if child(ren) use such games.

Phase: Pilot

Actively recruiting: Yes

Category: Adults, Remote (Online / Phone Call Only) Studies

SAR443579 in AML, B-ALL, or MDS

This study involves taking a study drug called SAR443579. The overall goal of this study is to find out if the study drug is safe and effective at treating adults and children with relapsed or refractory acute myeloid leukemia (R/R AML), high risk myelodysplastic syndrome (HR-MDS), or B-cell acute lymphoblastic leukemia (B-ALL). This is a first-in-human research study.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Pediatric Leukemias, Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL)

DCA Trial in PDC Deficiency

We are studying an investigational drug called sodium dichloroacetate (DCA). Investigational means that the study drug has not been approved by the US Food and Drug Administration (FDA) and that its use in this study is experimental. This a purpose of this research study is to determine whether DCA is a helpful treatment for patient with pyruvate dehydrogenase complex (PDC) deficiency (PDCD).

Phase: Phase III

Actively recruiting: No

Category: Children

The Charisma Study

We are currently enrolling adolescents ages 15-21 of South Asian ancestry, African American ancestry, and European American or "White" ancestry. This study is looking to better understand ancestry related differences in risks for developing cardiovascular disease and type 2 diabetes. The study involves 2 visits to the CHOP Main Hospital in Philadelphia, and participants will be compensated for their efforts.

Phase: N/A

Actively recruiting: No

Category: Adults, Children, Healthy Controls

Brains Keep Changing

We want to study how brain function and structure change in childhood. We will compare brain measures in children with and without autism spectrum disorder (ASD) who are between the ages of 10-13 years old.

Phase: Pilot

Actively recruiting: Yes

Category: Children, Healthy Controls, MRI/Imaging Studies

Healthy Controls EndoPAT

The purpose of this research study is to learn more about the normal function of blood vessels in healthy children and adolescents. This will help us discover differences between healthy individuals and children with lupus. Healthy control participants will be asked to make 1 visit to CHOP to complete a brief survey, a fasting blood draw and a test to measure the blood pressure in your fingers.

Phase: N/A

Actively recruiting: No

Category: Adults, Children, Healthy Controls, One-Time Visit Studies

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